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The cooperative international neuromuscular research group Duchenne natural history study: glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures.
Henricson EK, Abresch RT, Cnaan A, Hu F, Duong T, Arrieta A, Han J, Escolar DM, Florence JM, Clemens PR, Hoffman EP, McDonald CM; CINRG Investigators. Henricson EK, et al. Muscle Nerve. 2013 Jul;48(1):55-67. doi: 10.1002/mus.23808. Epub 2013 May 6. Muscle Nerve. 2013. PMID: 23649481 Free PMC article.
Development of a model-based clinical trial simulation platform to optimize the design of clinical trials for Duchenne muscular dystrophy.
Lingineni K, Aggarwal V, Morales JF, Conrado DJ, Corey D, Vong C, Burton J, Larkindale J, Romero K, Schmidt S, Kim S; Cooperative International Neuromuscular Research Group investigators and Duchenne Regulatory Science Consortium members. Lingineni K, et al. CPT Pharmacometrics Syst Pharmacol. 2022 Mar;11(3):318-332. doi: 10.1002/psp4.12753. Epub 2022 Jan 3. CPT Pharmacometrics Syst Pharmacol. 2022. PMID: 34877803 Free PMC article.
The cooperative international neuromuscular research group Duchenne natural history study--a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used.
McDonald CM, Henricson EK, Abresch RT, Han JJ, Escolar DM, Florence JM, Duong T, Arrieta A, Clemens PR, Hoffman EP, Cnaan A; Cinrg Investigators. McDonald CM, et al. Muscle Nerve. 2013 Jul;48(1):32-54. doi: 10.1002/mus.23807. Epub 2013 May 16. Muscle Nerve. 2013. PMID: 23677550 Free PMC article.
Intrathecal nusinersen treatment for SMA in a dedicated neuromuscular clinic: an example of multidisciplinary and integrated care.
Sansone VA, Albamonte E, Salmin F, Casiraghi J, Pirola A, Bettinelli M, Rao F, Mancini L, Tovaglieri N, Fedeli F, Stoia P, Heinen M, Cozzi V, Carraro E, Lunetta C, Di Bari A, Mercuri E; Italian EAP working group. Sansone VA, et al. Neurol Sci. 2019 Feb;40(2):327-332. doi: 10.1007/s10072-018-3622-9. Epub 2018 Nov 15. Neurol Sci. 2019. PMID: 30430317